WVE-N531 showed significant improvements in functional measures and muscle health in DMD patients during the phase 2 FORWARD-53 trial. Sustained exon skipping, dystrophin restoration, and improved ...

The researchers showed that the human muscle fibres connected to the nerves of the mice forming neuromuscular ... Muscular dystrophies are a group of genetic diseases that cause progressive weakness ...

We identified one particular microRNA, miR-486, as a muscle-enriched miRNA that is strongly reduced in expression levels in Duchenne muscular dystrophy (DMD), but not the milder Becker muscular ...

DMD, a genetic disorder affecting 1 in 5000 male births, is caused by mutations in the DMD gene. While it primarily leads to muscle wasting, a new paper published in Molecular Medicine highlights ...